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Finkel, Richard; Bertini, Enrico; Muntoni, Francesco; Mercuri, Eugenio
Neuromuscular disorders : NMD, 07/2015, Volume: 25, Issue: 7Journal Article, Conference Proceeding
Highlights • An updated classification for SMA is presented. • The utility and limitations of animal models in SMA is discussed. • Biomarkers that are potentially useful in a clinical trial setting are reviewed. • A detailed review of clinical outcome measures for use in a clinical trial is tabulated.
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